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Tyrosine Kinase Inhibitor

Asciminib for Pediatric Chronic Myeloid Leukemia

Phase 1 & 2

Recruiting

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female participants in the Pediatric formulation group must be ≥ 1 and less than 18 years of age at study entry. Male or female participants in the Adult formulation group must be ≥ 14 and less than 18 years of age with a body weight of ≥ 40 kg at study entry

Participants with Ph+ CML-CP must meet specific laboratory values at the screening visit including percentages of blasts, promyelocytes, basophils, neutrophils, platelet count, absence of extramedullary leukemic involvement, prior treatment with a minimum of one TKI, failure or intolerance to the most recent TKI therapy, specific performance status, adequate renal, hepatic, pancreatic, and cardiac function, electrolyte values within normal limits, and evidence of typical BCR-ABL1 transcript [e14a2 and/or e13a2] at the time of screening

Must not have

Known second chronic phase of CML after previous progression to AP/BC

History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up after first dose at week 1 day 1, 4 weeks, 52 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new cancer drug in children. The goal is to see if it works better than other drugs already being used to treat kids with cancer.

Who is the study for?

This trial is for pediatric patients aged 1 to <18 years with chronic myeloid leukemia who have been treated with at least one TKI. They must not be pregnant, have a history of severe liver disease or pancreatitis, and cannot plan for a stem cell transplant. Eligible participants should have stable blood counts and organ functions.

What is being tested?

The study tests two formulations of asciminib (pediatric and adult) in young patients previously treated with tyrosine kinase inhibitors (TKIs). It aims to establish the appropriate dose for children while assessing safety by comparing responses between age groups.

What are the potential side effects?

Potential side effects are not specified but may include those common to cancer treatments such as nausea, fatigue, changes in blood counts leading to increased infection risk, liver issues, and potential heart problems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My chronic myeloid leukemia has returned after worsening.

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I have had acute pancreatitis in the last year or have chronic pancreatitis.

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I have a history of liver disease.

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I have had a stem-cell transplant before.

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I am planning to have a stem cell transplant from a donor.

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I have a GI condition that affects how my body absorbs medication.

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I have a heart condition affecting its rhythm.

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My cancer has the T315I mutation.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ after first dose at week 1 day 1, 4 weeks, 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~after first dose at week 1 day 1, 4 weeks, 52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and after first dose at week 1 day 1, 4 weeks, 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Primary PK parameter: AUCtau

Primary Pharmacokinetic (PK) parameter: AUClast

Secondary PK parameter: Cmax

+2 more

Secondary study objectives

Hematologic responses

Molecular responses

Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: AsciminibExperimental Treatment2 Interventions

This arm consists of 2 groups: * The pediatric formulation group where the dose is based on body weight (1.3mg/kg BID or 2.6 mg/kg QD) * The adult formulation group where participants will receive a flat dose of 40mg BID

0 / 10Eligibility criteria met